Article Derived From Transcript of YouTube Video: My Quest to Cure Prion Disease — Before It’s Too Late | Sonia Vallabh | TED

Transcript of YouTube Video: My Quest to Cure Prion Disease — Before It’s Too Late | Sonia Vallabh | TED

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Transcript Summary

Sonia Vallabh shares her deeply personal journey of discovering her 50% genetic risk for prion disease, a fatal condition that took her mother's life. Despite not having a background in biomedical sciences, she and her husband Eric, motivated by the desire to understand and combat the disease, transitioned from their respective careers to pursue PhDs and lead a lab at the Broad Institute. Their mission is to develop a therapy for prion disease, focusing on prevention by targeting the PrP protein before it becomes pathogenic. Vallabh's story highlights the intersection of personal loss, scientific pursuit, and the quest for a cure, emphasizing the importance of audacity and humility in scientific exploration.

Detailed Transcript of YouTube Videos

The Liabilities of Leading a Life of Research

Leading this life has certain liabilities. Like ... things can get macabre if you Google search my name. And if you click on "obituary," dang. Bottom line, rumors of my death have been greatly exaggerated.

Understanding Prion Disease

OK, but let's talk about how prion disease works and what we need to do about it. Prion disease is unique in all of biology. The causal pathogen isn’t a virus, and it’s not a bacterium. It's this one normal protein called PrP that you normally have in your body.

The Misfolding Cascade

And it's normally not a problem, but it is capable of going rogue. And when it does, it changes shape. And then it goes around grabbing other copies of PrP, and it corrupts those. And this spreads through your brain and kills your neurons.

The Quest for a Therapy

Until recently, this was a process we could only infer. But now, thanks to state-of-the-art single-molecule imaging, we can observe it directly. Shown here at TED for the first time, I am so pleased to present to you the prion misfolding cascade in action.

A Different Approach to Treatment

When you look at the biology of this disease, any disease, where do your eyes go? They go to the train wreck. Right? Look at those scary rogue proteins. And if we think about how to treat this disease, we might think, go get those bad guys.

Prevention as the Goal

But Eric and I have come to see our mission differently. What if we can do the most good not by going after the big scary pathogens and lobbing fireballs at them, but instead by doing something much more understated and subtle?

The Challenge of Drug Development

We're lucky to have the series of clues from nature that indicate you can live a healthy life without PrP. So we’re scouring the globe for tools to dial it down. And brilliant ideas are an awesome start, but they also have to be wrangleable into actual, practical medicines.

The Importance of Timing in Treatment

And why get hung up on timing? Because your brain isn't any other organ. Your brain is what makes you you. Our greatest good isn't a drug that will stabilize me or anyone else mid-train wreck, one foot in the void.

The Paradox of Prevention

Where we have letters from the future to guide us, where what's at stake is irreplaceable human brains, we have to aim higher. We have to prevent.

The Unconventional Path of Clinical Trials

Come to find out, prevention isn't business as usual. Clinical trials are basically always done in sick patients post-train wreck. This is what's comfortable.

The Urgency of Our Mission

But we all know if you're having a heart attack and you walk into the ER at that moment and they give you a statin, it won't help. Prevention and treatment are different goals.

The Balance Between Boldness and Caution

I see this paradox at the heart of our mission. For sure, we are being summoned to be audacious. We know so much more about the brain and how to get drugs there than we did even a few years ago.

The Daily Reality of Scientific Research

So what is daily life like in the trenches? A decade ago, if you had asked me, "Sonia, what's the holy grail of your quest?" I would have said it's that molecule I told you about.

The Expanding Scope of Our Work

But what if finding the molecule isn't enough? It turns out to meaningfully test a new medicine in humans, especially for rare disease and especially for prevention, you need more, you need a lot more.

The Race Against Time

And if you're us, you need to be building it all in parallel because you are racing against the clock you can't see. So before our eyes, our scope has expanded from this ... to this. It's a lot.

The Journey and Its Outcomes

And maybe you're wondering how it's all going. Here's what I can say. There will be the race to the first drug and the race to the best drug. We’re far from the end of this quest, but we’re far from the beginning.

Embracing Uncertainty and Possibility

We don't have any guarantees. But what we do have, and gosh, are we lucky to have it, is jeopardy! There's more to say about what it's like to live with jeopardy, but as far as I can tell, at least, you all are human, and so I think on some level, you know.

Personal Reflection on Luck and Purpose

Recently, I told a friend that I consider myself lucky, and he gets all surprised. He's like, "Even with the mutation?" And my mind was kind of blown because this is me. There's no version of my life where you subtract the mutation and hold the rest constant.

The Gift of Life and Legacy

On the one hand, I got dealt a bad card. And don't get me wrong, I really don't want to die young. At the same time, this bad card has launched me on a quest with a team. And the wonder of this exact life is that I am constantly getting to meet people's best selves, including versions of Eric and me, that I wouldn't have encountered any other way.

The Role of Children in Our Journey

I want you to meet these guys. These are our kids. Daruka is the big one, Kavari is the also big one. We had them through IVF with preimplantation genetic testing to avoid passing on my mutation.

The Poetic Nature of Life

My mom never got to meet these kids, and she would have been a luminous grandma. But if she had, we wouldn't have known about my risk in time to avoid passing it on. So somewhere wrapped up in the grief of having lost her so young is this other thing, this transgenerational gift.

The Quest for a Cure Continues

I'm walking alongside these kids on their own journeys as best I can. And you know how it is with kids. Sometimes the shape of the future begs to be assumed. X number of years until Y. This parade of milestones. This storyboard. But here again is a luxury not all of us have.

The Call to Embrace Life's Uncertainty

And perhaps, in ways large and small, it's a luxury none of us have. What would it mean to do all of this less narratively? What if our lives, our lives together, are best lived not as prose, but as poetry? I'm still living into this question, but I'm glad to have it with me.

Closing Thoughts

Folks, thank you and wish us luck. We need it.

Notes

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